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The problem of therapeutic use of unauthorised (investigational) drugs, always controversial, over recent years has become a topic of a particularly heated public debate. Most often referred to as expanded access (EA) or compassionate use, such treatment is usually performed in patients with serious including life-threatening diseases who cannot be treated satisfactorily with authorised drugs.1 Remarkably, EA, originally introduced with a view to enabling treatment of patients with unauthorised drugs in exceptional cases and on a rather small scale, over the last decade has tremendously developed and at present is clearly of growing importance to public health.
According to the recently published US Food and Drug Administration’s (FDA’s) data, the number of EA requests in the USA increased more than twofold between 2005 and 2014; these applications pertain to use of unauthorised drugs in a variety of medical specialties.2 While analogous data for the European Union are lacking, in the majority of European countries EA treatment is permitted by drug regulatory agencies and at least in some of these countries the role of EA in clinical practice also tends to be rising.3
Particularly relevant to public health are EA programmes and their relations with clinical trials. Such programmes, often involving hundreds or even more than a …
Contributors All authors substantially contributed to the conception of the work. JB drafted the manuscript. AS, AB-H, MP, JR, AW-K and AG revised the manuscript critically for important intellectual content. All authors approved the final version of the manuscript.
Funding The authors have not declared a specific grant for this research from any funding agency in the public, commercial or not-for-profit sectors.
Disclaimer The views expressed in this article are those of the contributors and should not necessarily be interpreted as the position of the European Medicines Agency or any of its committees.
Competing interests AB-H is a member of the Compassionate Use Advisory Committee (CompAC), an external, expert panel of internationally recognised medical experts, bioethicists and patient representatives formed by NYU School of Medicine in collaboration with Janssen. CompAC advises Janssen about requests for compassionate use of its investigational medicines, and NYU receives administrative funding from Janssen to facilitate the CompAC Committee. Moreover, AB-H co-chairs the NYU Langone Health Working Group on Compassionate Use and Pre-Approval Access
Patient consent Not required.
Provenance and peer review Not commissioned; externally peer reviewed.